Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

Rosen, D. R., Siddique, T., Patterson, D., Figlewicz, D. A., Sapp, P., Hentati, A., Donaldson, D., Goto, J., O'Regan, J. P., Deng, H.-X., et al. (1993) · Nature · doi:10.1038/362059a0 · Cited by 7,062

Estimation of nuclear population from microtome sections

Abercrombie, M. (1946) · The Anatomical Record · doi:10.1002/ar.1090940210 · Cited by 4,363

Gene-expression profiles predict survival of patients with lung adenocarcinoma

Beer, D. G., Kardia, S. L. R., Huang, C.-C., Giordano, T. J., Levin, A. M., Misek, D. E., Lin, L., Chen, G., Gharib, T. G., Thomas, D. G., et al. (2002) · Nature Medicine · doi:10.1038/nm733 · Cited by 2,025

ALS-Linked SOD1 Mutant G85R Mediates Damage to Astrocytes and Promotes Rapidly Progressive Disease with SOD1-Containing Inclusions

Bruijn, L. I., Becher, M. W., Lee, M. K., Anderson, K. L., Jenkins, N. A., Copeland, N. G., Sisodia, S. S., Rothstein, J. D., Borchelt, D. R., Price, D. L., et al. (1997) · Neuron · doi:10.1016/s0896-6273(00)80272-x · Cited by 1,341

Local gene knockdown in the brain using viral-mediated RNA interference

Hommel, J. D., Sears, R. M., Georgescu, D., Simmons, D. L., & DiLeone, R. J. (2003) · Nature Medicine · doi:10.1038/nm964 · Cited by 334 · cited 1×

Decoding ALS: from genes to mechanism

Taylor, J. P., Brown, R. H., & Cleveland, D. W. (2016) · Nature · doi:10.1038/nature20413 · Cited by 2,066

Converging Mechanisms in ALS and FTD: Disrupted RNA and Protein Homeostasis

Ling, S.-C., Polymenidou, M., & Cleveland, D. W. (2013) · Neuron · doi:10.1016/j.neuron.2013.07.033 · Cited by 1,811

Onset and Progression in Inherited ALS Determined by Motor Neurons and Microglia

Boillée, S., Yamanaka, K., Lobsiger, C. S., Copeland, N. G., Jenkins, N. A., Kassiotis, G., Kollias, G., & Cleveland, D. W. (2006) · Science · doi:10.1126/science.1123511 · Cited by 1,623

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456

Molecular biology of amyotrophic lateral sclerosis: insights from genetics

Pasinelli, P., & Brown, R. H. (2006) · Nature Reviews Neuroscience · doi:10.1038/nrn1971 · Cited by 1,107

RNAi therapeutics: a potential new class of pharmaceutical drugs

Bumcrot, D., Manoharan, M., Koteliansky, V., & Sah, D. W. Y. (2006) · Nature Chemical Biology · doi:10.1038/nchembio839 · Cited by 1,085

Strategies for silencing human disease using RNA interference

Kim, D. H., & Rossi, J. J. (2007) · Nature Reviews Genetics · doi:10.1038/nrg2006 · Cited by 1,078

Non–cell autonomous toxicity in neurodegenerative disorders: ALS and beyond

Ilieva, H., Polymenidou, M., & Cleveland, D. W. (2009) · Journal of Cell Biology · doi:10.1083/jcb.200908164 · Cited by 1,008

Potential roles of gut microbiome and metabolites in modulating ALS in mice

Blacher, E., Bashiardes, S., Shapiro, H., Rothschild, D., Mor, U., Dori-Bachash, M., Kleimeyer, C., Moresi, C., Harnik, Y., Zur, M., et al. (2019) · Nature · doi:10.1038/s41586-019-1443-5 · Cited by 700

Neuroinflammation in amyotrophic lateral sclerosis: role of glial activation in motor neuron disease

Philips, T., & Robberecht, W. (2011) · The Lancet Neurology · doi:10.1016/s1474-4422(11)70015-1 · Cited by 646

Common and rare variant association analyses in amyotrophic lateral sclerosis identify 15 risk loci with distinct genetic architectures and neuron-specific biology

van Rheenen, W., van der Spek, R. A. A., Bakker, M. K., van Vugt, J. J. F. A., Hop, P. J., Zwamborn, R. A. J., de Klein, N., Westra, H.-J., Bakker, O. B., Deelen, P., et al. (2021) · Nature Genetics · doi:10.1038/s41588-021-00973-1 · Cited by 542

Antisense oligonucleotide therapy for neurodegenerative disease

Smith, R. A. (2006) · Journal of Clinical Investigation · doi:10.1172/jci25424 · Cited by 506

Drug Targeting to the Brain

Pardridge, W. M. (2007) · Pharmaceutical Research · doi:10.1007/s11095-007-9324-2 · Cited by 447

Glial cells as intrinsic components of non-cell-autonomous neurodegenerative disease

Lobsiger, C. S., & Cleveland, D. W. (2007) · Nature Neuroscience · doi:10.1038/nn1988 · Cited by 441

Paradoxical Roles of Antioxidant Enzymes: Basic Mechanisms and Health Implications

Lei, X. G., Zhu, J.-H., Cheng, W.-H., Bao, Y., Ho, Y.-S., Reddi, A. R., Holmgren, A., & Arnér, E. S. J. (2016) · Physiological Reviews · doi:10.1152/physrev.00010.2014 · Cited by 429

Astrocytes in Neurodegenerative Disease: Table 1.

Phatnani, H., & Maniatis, T. (2015) · Cold Spring Harbor Perspectives in Biology · doi:10.1101/cshperspect.a020628 · Cited by 420

Drug Targeting to the Brain

de Boer, A. G., & Gaillard, P. J. (2007) · Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.47.120505.105237 · Cited by 348

Recent Advances in Lentiviral Vector Development and Applications

Mátrai, J., Chuah, M. K., & VandenDriessche, T. (2010) · Molecular Therapy · doi:10.1038/mt.2009.319 · Cited by 347

Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice

Rodriguez-Lebron, E., Denovan-Wright, E. M., Nash, K., Lewin, A. S., & Mandel, R. J. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.05.006 · Cited by 275

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

Pfister, E. L., Kennington, L., Straubhaar, J., Wagh, S., Liu, W., DiFiglia, M., Landwehrmeyer, B., Vonsattel, J.-P., Zamore, P. D., & Aronin, N. (2009) · Current Biology · doi:10.1016/j.cub.2009.03.030 · Cited by 261

Blood–spinal cord barrier disruption contributes to early motor-neuron degeneration in ALS-model mice

Winkler, E. A., Sengillo, J. D., Sagare, A. P., Zhao, Z., Ma, Q., Zuniga, E., Wang, Y., Zhong, Z., Sullivan, J. S., Griffin, J. H., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1401595111 · Cited by 238

Amyotrophic lateral sclerosis: translating genetic discoveries into therapies

Akçimen, F., Lopez, E. R., Landers, J. E., Nath, A., Chiò, A., Chia, R., & Traynor, B. J. (2023) · Nature Reviews Genetics · doi:10.1038/s41576-023-00592-y · Cited by 234

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201

Activated protein C therapy slows ALS-like disease in mice by transcriptionally inhibiting SOD1 in motor neurons and microglia cells

Zhong, Z., Ilieva, H., Hallagan, L., Bell, R., Singh, I., Paquette, N., Thiyagarajan, M., Deane, R., Fernandez, J. A., Lane, S., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci38476 · Cited by 176

Amyotrophic Lateral Sclerosis: From Current Developments in the Laboratory to Clinical Implications

Cozzolino, M., Ferri, A., & Teresa Carrì, M. (2008) · Antioxidants & Redox Signaling · doi:10.1089/ars.2007.1760 · Cited by 156

Non-neuronal cells in amyotrophic lateral sclerosis — from pathogenesis to biomarkers

Vahsen, B. F., Gray, E., Thompson, A. G., Ansorge, O., Anthony, D. C., Cowley, S. A., Talbot, K., & Turner, M. R. (2021) · Nature Reviews Neurology · doi:10.1038/s41582-021-00487-8 · Cited by 147

PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model

Katsouri, L., Lim, Y. M., Blondrath, K., Eleftheriadou, I., Lombardero, L., Birch, A. M., Mirzaei, N., Irvine, E. E., Mazarakis, N. D., & Sastre, M. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1606171113 · Cited by 146

Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P. (2010) · Gene Therapy · doi:10.1038/gt.2009.119 · Cited by 124

Biomarkers in Neurodegenerative Diseases

Jeromin, A., & Bowser, R. (2017) · Advances in Neurobiology · doi:10.1007/978-3-319-57193-5_20 · Cited by 92

The role of altered protein acetylation in neurodegenerative disease

Kabir, F., Atkinson, R., Cook, A. L., Phipps, A. J., & King, A. E. (2023) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2022.1025473 · Cited by 75

Clinical Applications Involving CNS Gene Transfer

Kantor, B., McCown, T., Leone, P., & Gray, S. J. (2014) · Advances in Genetics · doi:10.1016/b978-0-12-800149-3.00002-0 · Cited by 74

Variability in SOD1-associated amyotrophic lateral sclerosis: geographic patterns, clinical heterogeneity, molecular alterations, and therapeutic implications

Huang, M., Liu, Y. U., Yao, X., Qin, D., & Su, H. (2024) · Translational Neurodegeneration · doi:10.1186/s40035-024-00416-x · Cited by 64

Amyotrophic lateral sclerosis pathogenesis: a journey through the secretory pathway

Nassif, M., Matus, S., Castillo, K., & Hetz, C. (2010) · Antioxidants & Redox Signaling · doi:10.1089/ars.2009.2991 · Cited by 60

Efficient gene expression from integration-deficient lentiviral vectors in the spinal cord

Peluffo, H., Foster, E., Ahmed, S. G., Lago, N., Hutson, T. H., Moon, L., Yip, P., Wanisch, K., Caraballo-Miralles, V, Olmos, G., et al. (2013) · Gene Therapy · doi:10.1038/gt.2012.78 · Cited by 40

Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

Tosolini, A. P., & Sleigh, J. N. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00129 · Cited by 32

Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?

Gherardini, L., Bardi, G., Gennaro, M., & Pizzorusso, T. (2014) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-013-1310-8 · Cited by 28

AAV-NRIP gene therapy ameliorates motor neuron degeneration and muscle atrophy in ALS model mice

Chen, H.-H., Yeo, H.-T., Huang, Y.-H., Tsai, L.-K., Lai, H.-J., Tsao, Y.-P., & Chen, S.-L. (2024) · Skeletal Muscle · doi:10.1186/s13395-024-00349-z · Cited by 14